NL-OMON52516招募中3 期
A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping - MIS51O
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 15（—）
入选标准
- •Inclusion Criteria:
- •Be a male with an established clinical diagnosis of DMD and an out-of-frame
- •deletion mutation of the DMD gene amenable to exon 51 skipping.
- •Ambulatory participant, able to perform TTRISE in 10 seconds or less at the
- •time of screening visit.
- •Able to walk independently without assistive devices.
- •Have intact right and left biceps muscles or an alternative upper arm muscle
- •Have been on a stable dose or dose equivalent of oral corticosteroids for at
- •least 12 weeks prior to randomization and the dose is expected to remain
- •constant (except for modifications to accommodate changes in weight and
- •stress-related needs as per the recently published guidelines throughout the
- •For ages 7 years and older, has stable pulmonary function (forced vital
- •capacity >=50 percent (%) of predicted and no requirement for nocturnal
- •ventilation). For ages 4 to 6 years, does not require support from ventilator
- •or non-invasive ventilation at time of screening.
排除标准
- •Exclusion Criteria:
- •Use of any pharmacologic treatment (other than corticosteroids) within 12
- •weeks prior to randomization.
- •Current or previous treatment with any other experimental pharmacologic
- •treatment for DMD or any prior exposure to antisense oligonucleotide, gene
- •therapy or gene editing; except the following: Ezutromid in the last 12 weeks
- •prior to first dose; Drisapersen in the last 36 weeks prior to first dose;
- •Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12
- •weeks prior to first dose; and Eteplirsen (previous or current use).
- •Major surgery within 3 months prior to randomization.
- •Presence of any other significant neuromuscular or genetic disease other than
- •Presence of any known impairment of renal function and/or other clinically
- •significant illness.
- •Has evidence of cardiomyopathy, as defined by left ventricular ejection
- •fraction less than <50% on the screening echocardiogram or Fridericia's
- •correction formula (QTcF) >=450 millisecond based on the screening
- •electrocardiograms (ECGs).
- •Other inclusion/exclusion criteria apply.
研究者
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